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Stem Cells Transl Med. 2016 Sep 13. pii: sctm.2015-0427. [Epub ahead of print]

Concise Review: Mesenchymal Stem Cell Therapy for Pediatric Disease: Perspectives on Success and Potential Improvements.

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  • 1Division of Neonatology, Rainbow Babies and Children's Hospital, Cleveland, Ohio, USA.
  • 2Division of Pulmonology, Rainbow Babies and Children's Hospital, Cleveland, Ohio, USA Department of Pediatrics, Case Western Reserve University, Cleveland, Ohio, USA


: Mesenchymal stem cells (MSCs) represent a potentially revolutionary therapy for a wide variety of pediatric diseases, but the optimal cell-based therapeutics for such diversity have not yet been specified. The published clinical trials for pediatric pulmonary, cardiac, orthopedic, endocrine, neurologic, and hematologic diseases provide evidence that MSCs are indeed efficacious, but the significant heterogeneity in therapeutic approaches between studies raises new questions. The purpose of this review is to stimulate new preclinical and clinical trials to investigate these factors. First, we discuss recent clinical trials for pediatric diseases studying MSCs obtained from bone marrow, umbilical cord and umbilical cord blood, placenta, amniotic fluid, and adipose tissue. We then identify factors, some unique to pediatrics, which must be examined to optimize therapeutic efficacy, including route of administration, dose, timing of administration, the role of ex vivo differentiation, cell culture techniques, donor factors, host factors, and the immunologic implications of allogeneic therapy. Finally, we discuss some of the practicalities of bringing cell-based therapy into the clinic, including regulatory and manufacturing considerations. The aim of this review is to inform future studies seeking to maximize therapeutic efficacy for each disease and for each patient.


Mesenchymal stem cells (MSCs) are the focus of great excitement for treating diseases associated with not just regeneration but also immunomodulation. This review focuses on the outcomes of MSC therapeutics in a variety of pediatric diseases. The discussion is based on how the trials took place and what can ultimately be learned from the outcomes of the studies. This review provides significant insight into learning the next steps toward developing better therapies for children with difficult-to-treat diseases.


Autism; Bronchopulmonary dysplasia; Graft versus host disease; Mesenchymal stem cells; Osteogenesis imperfecta; Pediatric diseases

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